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Lip and Oral Cavity CancerPhase 1/2NCT00064103

Gene Therapy in Preventing Cancer in Patients With Premalignant Carcinoma of the Oral Cavity or Pharynx

A Phase 1/2 study testing Ad5CMV-p53 gene and laboratory biomarker analysis in people with Lip and Oral Cavity Cancer and Oropharyngeal Cancer, run by National Cancer Institute (NCI) at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This phase I/II trial is studying the side effects and best dose of gene therapy and to see how well it works in preventing cancer in patients with premalignant carcinoma of the oral cavity or pharynx. Inserting the p53 gene into a person's tumor cells may improve the body's ability to kill the tumor cells Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Ad5CMV-p53 gene and laboratory biomarker analysis
Could I receive a placebo?
No, everyone receives an active treatment
How many people
51 participants
Where
1 siteUnited States
Study dates
Jun 2003to Aug 2007
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Histologically confirmed mild to moderate dysplasia OR severe dysplasia/carcinoma in situ of the oral cavity or oral pharynx
  • Clinically evident diffuse premalignant disease, defined by 1 of the following mucosal abnormalities:
  • Extension between adjacent organ structures (e.g., lateral tongue, ventral tongue, and the floor of the mouth)
  • Extensive surface area, including the entire ventral tongue or floor of the mouth or buccal mucosa, in a velvety "indiscreet" pattern
  • Meets 1 of the following criteria:
  • Previously treated with conventional treatment (e.g., radiotherapy or surgery) for a prior head and neck malignancy
Show all 37Show fewer
  • Failed biochemoprevention approaches for premalignant disease
  • Failed other therapeutic approaches for premalignant disease
  • No active squamous cell carcinoma of the head and neck
  • Performance status - Karnofsky 70-100%
  • Absolute granulocyte count at least 2,000/mm^3
  • Platelet count at least 100,000/mm^3
  • Bilirubin no greater than 1.0 mg/dL
  • Creatinine no greater than 1.5 mg/dL
  • No hypertension (baseline blood pressure 140/90 mm Hg or higher)
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective barrier contraception during and for 1 year after study participation
  • HIV-1 negative
  • No known contact with former tissue or organ transplantation recipients or individuals with severe immunodeficiency disease (acquired or congenital) during and for 28 days after study treatment
  • No prior malignancy within the past 2 years except nonmelanoma skin cancer or aerodigestive cancer
  • No active systemic viral, bacterial, or fungal infections requiring treatment
  • No serious concurrent illness that would preclude study compliance and follow-up
  • No psychological, familial, sociological, geographical, or other condition that would preclude study compliance and follow-up
  • See Disease Characteristics
  • More than 21 days since prior chemotherapy (42 days for mitomycin and nitrosoureas)
  • No concurrent systemic chemotherapy
  • No concurrent prednisone or the equivalent, including corticosteroids of more than 10 mg/day
  • See Disease Characteristics
  • More than 3 months since prior radiotherapy involving the lesion selected for this study
  • No concurrent radiotherapy
  • See Disease Characteristics
  • More than 8 weeks since prior investigational agents
  • No prior experimental therapy (i.e., oral, systemic, topical, or direct injection) for the lesion selected for treatment in this study
  • No other concurrent immunosuppressive therapy
  • No other concurrent investigational agents
  • No concurrent aspirin dose greater than 175 mg/day

Who cannot join

None listed.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • M D Anderson Cancer Center

    Houston, Texas, 77030

Other trials in United States

About this study

Official title: Clinical Protocol for Wild Type p53 Gene Induction in Premalignancies of Squamous Epithelium of the Oral Cavity and Oral Pharynx Via an Adenoviral Vector [NCI Supplied Agent Ad-p53, (INGN 201) (Advexin®) NSC 683550, IND# 7135]

This phase I/II trial is studying the side effects and best dose of gene therapy and to see how well it works in preventing cancer in patients with premalignant carcinoma of the oral cavity or pharynx. Inserting the p53 gene into a person's tumor cells may improve the body's ability to kill the tumor cells

Read the detailed description

OBJECTIVES:

  1. Determine the acute toxic effects of Ad5CMV-p53 gene administered as an oral rinse and as an intramucosal injection in patients with diffuse premalignant carcinoma of the oral cavity or oral pharynx.

II. Determine the maximum tolerated dose of this drug in these patients. III. Determine the topical transduction efficiency of adenoviral-mediated wild type p53 gene transfer in patients treated with this drug.

IV. Determine the efficacy of this drug in reversing the histology of oral premalignancies in these patients.

  1. Determine the distribution of transgenic protein within the area of the premalignant lesion in patients treated with this drug.

OUTLINE: This is an open-label, dose-escalation study of Ad5CMV-p53 gene administered as an oral rinse.

Phase I: Patients receive Ad5CMV-p53 gene by intramucosal injection into the area of the lesion followed at least 2 hours later by Ad5CMV-p53 gene as an oral rinse on day 1. Patients then receive Ad5CMV-p53 gene as an oral rinse twice daily on days 2-5. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity. Cohorts of 3-6 patients receive escalating doses of Ad5CMV-p53 gene as an oral rinse until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.

Phase II: Patients receive treatment with intramucosal Ad5CMV-p53 gene as in phase I and Ad5CMV-p53 gene as an oral rinse at the MTD. Patients are followed every 3 months for 1 year, every 6 months for 1 year, and then annually for 3 years. Patients then receive long-term follow-up annually for an additional 10 years.

Being tested
Ad5CMV-p53 gene, laboratory biomarker analysis
Medical subject headings
Mouth Neoplasms, Oropharyngeal Neoplasms, Tongue Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Ad5CMV-p53 geneBiological

    Also called Ad5CMV-p53, ADVEXIN, INGN-201

    Given intramucosally or as oral rinse

  • laboratory biomarker analysisOther

    Correlative studies

Study groups in NCT00064103
Study groupRoleWhat they receive
Treatment (Ad5CMV-p53 gene)ExperimentalPhase I: Patients receive Ad5CMV-p53 gene by intramucosal injection into the area of the lesion followed at least 2 hours later by Ad5CMV-p53 gene as an oral rinse on day 1. Patients then receive Ad5CMV-p53 gene as an oral rinse twice daily on days 2-5. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity. Cohorts of 3-6 patients receive escalating doses of Ad5CMV-p53 gene as an oral rinse until the MTD is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity. Phase II: Patients receive treatment with intramucosal Ad5CMV-p53 gene as in phase I and Ad5CMV-p53 gene as an oral rinse at the MTD.
Receives: Biological: Ad5CMV-p53 gene; Other: laboratory biomarker analysis

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Frequency of adverse events

    Measured: Up to 15 years

  2. Severity of adverse events graded using the CTCAE version 3.0

    Measured: Up to 15 years

  3. Maximum tolerated dose of Ad5CMV-p53 gene

    Evaluated by the frequency and relationship of dose-limiting toxicities, if any, experienced by patients during dose escalation.

    Measured: 6 months

  4. Pharmacodynamics evaluated by examining the injected precancerous lesion for induction of apoptosis and expression of the p53 protein

    Presented using descriptive statistics, frequency tabulations, and graphical displays over time by treatment cohort.

    Measured: 168 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Jul 2003 (estimated)
  2. Study startJun 2003
  3. Primary completionAug 2007

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Prevention
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
51 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00064103
Sponsor's protocol ID
NCI-2012-02541
Other IDs
MDA-ID-00193, P50CA097007, CDR0000306522
Status last verified
Jan 2013
First submitted
8 Jul 2003
Last updated
23 Jan 2013
Results posted
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Ad5CMV-p53 gene and laboratory biomarker analysis in people with Lip and Oral Cavity Cancer and Oropharyngeal Cancer, run by National Cancer Institute (NCI) at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 37 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 23 Jan 2013. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Frequency of adverse events (time frame: Up to 15 years). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00064103.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jan 2013.

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